Patient & Family Resources

It is reported that one in 200 Americans will receive a stem cell transplant in their lifetime. Only 30 percent of patients will find a matching donor within their families, with 70 percent seeking for unrelated matching donors in the bone marrow registry. 

A diagnosis of a life-threatening disease such as leukemia or lymphoma can be emotionally devastating. Often patients and their loved ones don’t know what to do first or where to turn for help. The resources presented here are intended to provide information on first steps for newly diagnosed patients whose healthcare professionals advise them that they will need a bone marrow or blood stem cell transplant.

Becoming well-informed makes it easier to take charge and advocate for yourself or a loved one.

What is a transplant?

A transplant is an infusion of healthy blood stem cells into the body to replace the ones producing the disease. Transplants can be autologous (aw-TAWL-uh-gus) meaning cells come from the patients themselves, or allogeneic (AL-oh-jen-EE-ik) meaning cells donated by another person. The type of cells used are blood stem cells, the immature cells that can grow into all types of blood cells. These stem cells may be found in three places:

• Within the bone marrow – a soft tissue found inside some of the bones, including large amounts in the bone of the hip and thigh – that creates stem cells and blood cells
• Circulating in small numbers within the blood itself
• In cord blood, the blood found inside the umbilical cord after a baby’s birth

The transplant collects these stem cells and infuses them into the recipient, where they migrate on their own into the bone marrow and begin the work of creating new blood cells and immune system.

What conditions are helped by transplantation?

Conditions that can be treated by transplant include:

• Bone marrow diseases: Amegakaryocytosis/congenital thrombocytopenia, Fanconi anemia, Paroxysmal nocturnal hemoglobinuria (PNH), Pure red cell aplasia, Severe aplastic anemia. In these diseases the bone marrow does not function properly.
   
• Familial erythrophagocythic lymphohistiocytosis and other histiocytic disorders.
   
• Hemoglobinopathies: Beta Thalassemia Major and Sickle Cell Disease. In these diseases the red blood cells do not function properly.
   
• Inherited Immune System Disorders: Severe combined immunodeficiency and Wiskott-Aldrich syndrome.
   
• Inherited Metabolic Disorders: Adrenoleukodystrophy (ALD), Hurler Syndrome (MPS-IH), Krabbe Disease (GLD), and Metachromatic Leukodystrophy (MLD).
   
• Leukemias and lymphomas: Acute Lymphoblastic Leukemia (ALL), Acute Myelogenous Leukemia (AML), Chronic Lymphocytic Leukemia (CLL), Chronic Myelogenous Leukemia (CML), Hodgkin’s (HL) and Non-Hodgkin’s Lymphoma (NHL), and Juvenile Myelomonocytic Leukemia.
   
• Myelodysplastic syndromes including myeloproliferative disorders.
   
• Multiple myeloma and plasma cell disorders.
   
• Some other cancers and malignant diseases.

What are the first steps after diagnosis?

If you are diagnosed with a condition that is treatable with a transplant, you should schedule a consultation with a transplant center as soon as possible. Your physician will explain your treatment options and recommend the most appropriate type of transplant for you. If time permits, many patients seek out a second opinion about treatment. 

Selecting a hospital

When possible, patients are encouraged to obtain more than one opinion before choosing a hospital for treatment. Larger centers have cancer research programs in clinical, basic science, and prevention (population-based investigations) that are designated as comprehensive cancer centers by the National Cancer Institute. This ensures a strong body of interactive research leading to advances in patient treatments.

Patients have many different reasons for choosing specific transplant centers. The major considerations include:

• Physician and Personal Referrals   
• Geographic Constraints
• Family Limitations
• Insurance Coverage/Financial Constraints
• Disease-Specific Specialization
• Personal Preference
• Professional Reputation 

Continue to next section, Finding a Matching Donor.